The Food and Drug Administration (FDA) has granted Orphan Drug designation to AMT-101 for the treatment of pouchitis, an inflammation that develops following ileal pouch-anal anastomosis surgery in patients with ulcerative colitis.
AMT-101 is a novel, gastrointestinal-selective, oral recombinant biologic fusion protein of human interleukin (IL)-10. Through proprietary technology, the investigational agent is able to cross the intestinal epithelial barrier allowing it to interact directly with the primary site of inflammation.
The designation is supported by data from the 12-week, randomized, double-blind phase 2 FILLMORE trial (ClinicalTrials.gov Identifier: NCT04741087), which evaluated the efficacy and safety of AMT-101 administered orally as monotherapy in 22 adults with chronic antibiotic-resistant pouchitis (modified Pouchitis Disease Activity Index score ≥5). Study participants also had a median baseline Geboes score of 5.1, representing severe pouchitis with ulceration and tissue destruction.
The primary endpoint was stool frequency response (defined as a reduction of ≥3 stools and ≥30% from baseline, or ≤ post-colectomy normal). Histologic response, defined as a Geboes score ≤3.1, was designated as a secondary outcome measure.
Results showed that 36.4% (n=8/22) and 22.7% (n=5/22) of patients treated with AMT-101 achieved stool frequency response and histologic response, respectively. AMT-101 was reported to be safe and well tolerated with mostly mild to moderate treatment emergent adverse events.
“Pouchitis is a severe end-stage complication of ulcerative colitis with serious symptoms that have a tremendous negative impact on quality of life, including excessive stool frequency, urgency, fecal incontinence and chronic pain,” said Bittoo Kanwar, MD, chief medical officer of AMT. “Following the results observed in our FILLMORE trial demonstrating compelling activity of AMT-101 in pouchitis, we look forward to continued collaboration with the FDA as we remain focused on phase 3 advancement in this patient population.”
Applied Molecular Transport announces FDA Orphan Drug designation granted to AMT-101 for treatment of pouchitis. News release. Applied Molecular Transport. Accessed November 10, 2022. https://www.globenewswire.com/news-release/2022/11/10/2553132/0/en/Applied-Molecular-Transport-Announces-FDA-Orphan-Drug-Designation-Granted-to-AMT-101-for-Treatment-of-Pouchitis.html